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Active trials

23 of 152 total trials

A Clinical Efficacy and Safety Study of OHB-607 in Preventing Bronchopulmonary Dysplasia in Extremely Premature Infants

The purpose of this study is to determine if an investigational drug can prevent Bronchopulmonary Dysplasia, reducing the burden of chronic lung dise...

Chronic Lung Disease of Prematurity
Intraventricular Hemorrhage
Drug: OHB-607

The purpose of the study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of VX-670 at different single and multiple d...

Myotonic Dystrophy Type 1 (DM1)
Drug: Placebo
Drug: VX-670

A phase 2 randomized, double blind, placebo controlled study evaluating the efficacy and safety of AL002 in participants with Early Alzheimer's Disea...

Active, not recruiting
Alzheimer Disease
Drug: Placebo
Drug: AL002

A Phase 2 open label study evaluating the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) of AL001 in participants with a Granu...

Active, not recruiting
Frontotemporal Dementia
Drug: AL001

A phase 3 double blind, placebo controlled study evaluating the efficacy and safety of AL001 in participants at risk for or with frontotemporal demen...

Active, not recruiting
Frontotemporal Dementia
Drug: Open label - AL001
Drug: AL001

The primary purpose of this study is to compare the effect of mitapivat versus placebo on anemia in participants with alpha- or beta-non-transfusion...

Active, not recruiting
Non-Transfusion-dependent Alpha-Thalassemia
Non-Transfusion-dependent Beta-Thalassemia
Drug: Placebo Matching Mitapivat
Drug: Mitapivat

The primary purpose of this study is to compare the effect of mitapivat versus placebo on transfusion burden in participants with transfusion-depende...

Active, not recruiting
Transfusion-dependent Alpha-Thalassemia
Transfusion-dependent Beta-Thalassemia
Drug: Placebo Matching Mitapivat
Drug: Mitapivat

The purpose of this study is to determine if treatment with apalutamide plus androgen deprivation therapy (ADT) before and after radical prostatectom...

Active, not recruiting
Prostatic Neoplasms
Drug: Placebo
Drug: Apalutamide

This Phase IIb study is a two part, multicenter study to evaluate the efficacy, safety, pharmacokinetics and pharmacodynamics of ATL1102 in non-ambul...

Active, not recruiting
Duchenne Muscular Dystrophy
Drug: Placebo
Drug: ATL1102 50mg

This Phase 3, 2-arm, randomized, active comparator-controlled, open-label, multicenter study will compare the efficacy and health-related quality of...

Active, not recruiting
Multiple Myeloma
Drug: Selinexor
Drug: Dexamethasone

Continuation study to provide continued access to latozinemab for participants who have previously participated in a latozinemab study

Neurodegenerative Diseases
Drug: Latozinemab

This is a multi-center randomized, double-blind, placebo-controlled, parallel, 4-arm study of nalbuphine ER (NAL ER).After meeting eligibility during...

Idiopathic Pulmonary Fibrosis
Drug: nalbuphine ER 54 mg
Drug: nalbuphine ER 27 mg

Part A of this trial will evaluate the safety and tolerability of a single unilateral administration of one of two dose levels of AAVAnc80-hOTOF and...

Sensorineural Hearing Loss, Bilateral
Combination Product: AAVAnc80-hOTOF via Akouos Delivery Device

The object of this trial is to evaluate whether the introduction of a targeted therapy after 4 cycles of the current standard-of-care treatment for a...

Biliary Tract Neoplasms
Drug: Gemcitabine
Drug: Niraparib

A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Explora...

Muscular Dystrophy, Landouzy Dejerine
Fascioscapulohumeral Muscular Dystrophy
Drug: AOC 1020
Drug: Placebo

A prospective, randomized, double-blind, placebo controlled, multi-center therapeutic study for patients age 3 and older with confirmed diagnosis of...

Active, not recruiting
Niemann-Pick Disease, Type C1
Drug: Placebo
Drug: Hydroxypropyl-beta-cyclodextrin

The goal of this clinical trial is to compare the safety and efficacy of AON-D21 versus placebo, both on top of standard of care, in patients with se...

Community-acquired Pneumonia
Drug: AON-D21
Drug: Placebo

Evaluate the safety and efficacy of oral azacitidne (CC-486) twice daily (BID) in subjects with myelodysplastic syndromes who failed to achieve an ob...

Active, not recruiting
Myelodysplastic Syndromes
Drug: Durvalumab
Drug: Oral Azacitidine

The purpose of this trial is to investigate the safety and efficacy of acasunlimab (also known as GEN1046) as monotherapy and in combination with pem...

Active, not recruiting
Non Small Cell Lung Cancer Metastatic
Biological: Pembrolizumab
Biological: Acasunlimab

This is a prospective, multinational, multicentre, randomised, parallel-group, open-label study to assess the safety, tolerability and performance of...

Begins enrollment this month
Respiratory Failure
Device: NucleoCapture device

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